Safety and Efficacy of LX102 Gene Therapy in Patients With Neovascular Age-related Macular Degeneration (nAMD) (VENUS)

A Phase 2, Randomized Controlled, Open-Label Study to Establish the Safety and Efficacy of LX102 in Patients With Neovascular Age-related Macular Degeneration (nAMD) (VENUS)

The goal of this study is to evaluate the overall safety and efficacy of LX102 gene therapy for nAMD.

In this Phase 2, multi-center, randomized controlled study, subjects will be randomized to receive one of the two dose levels of LX102 (n=20 for each dose level), or aflibercept (n=10).

Safety, tolerability, and efficacy will be evaluated for a period of approximately 1 year from baseline.

Inclusion Criteria:

1. Willing to sign the informed consent, and willing to attend follow-up visits.
2. Age ≥ 50, and ≤ 89.
3. Diagnosis of active CNV secondary to neovascular AMD.
4. BCVA ETDRS letters between 19 and 73.
5. Demonstrated a meaningful response to anti-VEGF therapy.

Exclusion Criteria:

1. CNV or macular edema in the study eye secondary to diseases other than nAMD.
2. Retinal detachment, uveitis, uncontrolled glaucoma in the study eye, or any condition preventing visual acuity improvement.
3. Absence of RPE tear at Screening.
4. Acute coronary syndrome, myocardial infarction or coronary artery revascularization, CVA, TIA in the last 6 months.
5. Uncontrolled hypertension defined as average SBP ≥160 mmHg or an average DBP ≥100 mmHg.
6. Uncontrolled diabetes defined as HbA1c >8.0%.